In this article I am going to ignore the typical metrics that are cited, such as the importance of clinical trial data being entered into the database, time to market and the cost of patient exclusivity. Instead, I shall focus on the influence of developing a truly patient-focused clinical trial, and the impact this has on the patient and their families. Whilst this is an aim we ought to strive for in all trials, it is especially important in the field of oncology studies, and even more so when the patient is terminally ill with a forecasted shortened life expectancy.
Instead of offering theoretical examples, I shall summarise actual real-life scenarios. These include the option for the patient of having visits at a location convenient for them. The traditional site is not completely replaced. Instead, having options really can have a positive impact on their lives and the lives of their families. Patients feel empowered and motivated to join or continue in their clinical trial.
What is Meant by a Truly Patient-focused Clinical Trial?
In my opinion, the definition of being truly patient-centric is simply allowing a patient to participate in a clinical trial with minimal disruption to their personal and family lives. How this might look in practice will depend upon the indication itself. Within the realm of oncology, this would be taking the visit to the patient’s home. In other areas it might be at school or at work.
This approach means taking the clinic to the patient and allowing the patient to build an optimum relationship with their visiting study nurse. This is often the same nurse throughout, thus giving the nurse a broader view of that patient’s wider health. This approach also enables the involvement of individuals that live in more remote locations to participate. They may previously have thought the distance to clinic would be too challenging. This type of service alleviates stress and improves the overall patient experience in studies where enrolment is typically an issue. A good example is in rare disease studies. Here sites can be few in number, indication-led issues a major issue, and often with substantial travel to site, in extreme cases even to a different country.
Typically, this approach has been successful in indications where enrolment has been challenging. That said, I feel the option should be offered more widely. Oncology trials have historically recruited well with patients driven by looking for a cure and supporting that effort. However, as you’ll see below, there is great human benefit from being able take the trial to the patient in these instances. In my view it makes perfect sense to go to the patient at times when their immune system is vulnerable, and they are feeling physically weaker than usual.
This approach to clinical trials generally enhances recruitment rates as patients need to visit the site less regularly. It also enables those less mobile or who live further away to participate when they otherwise might not enter a clinical trial. Research nursing can also improve retention rates as patients build an open relationship with their visiting nurse. These factors provide extra support to the patient, whilst also enabling the drug to potentially reach the marketplace more quickly.
Example 1 - "Sheila"
The first situation involves a female patient with children and grandchildren who enrolled into a clinical trial for subjects with terminal ovarian cancer. I shall call this lady Sheila, for the sake of this article. As you would expect, Sheila became tired very easily and found trips to the site for visits difficult. Clearly, for anyone facing a terminal diagnosis, time is a very precious commodity. It is heartening to see people participate in clinical trials when there is clearly no gain for them personally. It is worth noting though that they consent to take part in trials of new drugs for the benefit of others in future generations. It is incumbent upon us all working in the clinical trials field to ensure that the burden on these patients is minimal.
This Phase I, first-in-human trial involved the patient travelling to site every day Monday to Friday to receive a subcutaneous injection. This was extremely onerous for the patient and family if they did not live in a short radius to the hospital site. Having to travel and park could mean the hospital appointment time would take several hours, if not the whole of the patient’s day! Following regulatory approval, it was agreed with the PI that following cycle 1 of the investigational medicinal product (IMP), the patient could receive the IMP off-site Tuesday to Friday, meaning that only one visit per week on a Monday had to be conducted at site. The IMP had to be administered under temperature-controlled conditions and transported at 2–8 degrees Celsius.
During one of the home visits, Sheila explained to our mobile research nurse the real impact this had on her in her final months of life. Every year in December, Sheila would make a Christmas pudding for each of her five grown-up children. It was a family tradition and extremely important to them all. Sheila laughed and said that it seems like such a trivial thing but for someone facing the end of their life with limited time left, the Christmas pudding ritual became extremely significant to them. Our nurse relayed what she had said: “If I had needed to travel to the hospital for all of these trial visits, I simply would not have had the energy left to prepare the Christmas puddings when I got home. Allowing the flexibility for me to be at home has made it possible for me to do this one last time for my children.”
Example 2 - "Karen"
Karen This example features another patient from the trial outlined above; here we have named her Karen. Karen had found the hospital experience very challenging and admitted to her research nurse she had thought about dropping out previously if this service had not have been available.
The demands of the hospital visits were becoming too much. The daily travel and time away from her family, which was often up to seven hours a day, were causing great distress. Karen’s husband was struggling to juggle the lengthy visits and time at the hospital with maintaining some sort of normal family life. This was also causing Karen great distress as she was labelling her visits a burden on her family.
Karen also had a needle phobia – not uncommon – but Karen’s was significant and heightened by the stress of being in a clinical environment. The waiting around, knowing the procedure would be happening but not quite knowing when, made it worse.
Research nursing at home really benefitted Karen. The same nurse was able to visit Karen each day, which reduced Karen’s anxiety levels dramatically, making the required visit procedures easier to perform. Karen also reported feeling much more relaxed as the routine of the same nurse attending in a pattern had eased her anxiety. Karen built an excellent rapport with her visiting research nurse and she felt better able to ask any questions she had about the trial or drug.
Example 3 - "Emily"
Another example of tailoring a trial to suit the subject is that of Emily (not her real name), aged 7. Emily had cancer and was enrolled into a clinical trial for a new chemotherapy agent.
Not unsurprisingly, Emily hated going to the hospital. The sterile, unfamiliar environment scared her. Even the thought of our mobile research nurse coming to the home to administer the chemotherapy was frightening. However, Emily loved fairies. At the next scheduled home visit, the family opened the door to our nurse and Emily was delighted to see that she was dressed in a fairy outfit! This made the visits more palatable and Emily felt slightly more in control of the situation. As our nurse recalled, “our paediatric patient would happily sit while I gave her intravenous chemotherapy as long as I was dressed as a fairy complete with tiara and wings! She said that I was her ‘get better fairy’ and that the trial medication was ‘magic potion’. I was very happy to comply. Home visits result in happier parents and increased compliance.”
Attention to detail and rapport is everything. If the nurse can speak to the child on their level, it makes for a more relaxed environment. For Emily, being surrounded by her family, pets and a nurse dressed as a fairy while having the chemotherapy reduced her stress levels and made her participation in the trial far less unsettling.
Since then, our nurses carry cuddly teddy bears dressed up as nurses when they visit children at home. Performing role-play using the bears makes explaining study procedures more child-friendly and less intimidating.
The sponsor benefits too...
Although our primary focus here is the patient, sponsors are vital in ensuring this sort of service can be made available to patients and their appreciation of the added value to the patient is vital. The sponsor of Sheila and Karen’s trial commented:
“The nurses were incredibly flexible, allowing patients to choose the time of the visit to fit around their daily activities of living, family and home life... Involving research home nursing improved the quality of life for those patients. At the end of life, enrolling in a trial is stressful, but with this patient-centric approach, it is a less stressful experience and helps to enrol and retain research patients when they know that this resource is available to them.”
In closing… These three examples show that, by offering a more patient-focused approach to participation in clinical trials, one can make a significant, positive impact on patients and families.
Many people facing a terminal diagnosis will decide for the good of future generations to take part in studies of new drugs, whether or not it will help them directly. However, this does not mean that when designing these important oncology studies, we do not take time to consider and ensure the subject is not put under undue strain because of their involvement. It is only by the goodwill of the patient we can move forward with this pioneering vital research. By designing trials to ease the burden on the patients, this in itself has a positive effect on the very metrics that industry typically concentrate on, like patient enrolment and retention rates. Faster enrolment and a reduction in the dropout of patients subsequently allows for a drug study to close quicker and to reach the marketplace potentially faster than another compound. This is when the value of a truly patient-centric approach is most obvious. Finally, it is noteworthy that no less than 85% of all clinical trials fail to recruit enough patients. 80% are delayed due to recruitment problems. Even then dropout rates can be prohibitively high. Source biopharmadive.com.
It is not an exaggeration to state that the current situation cannot continue. Resources are indeed finite. This then is one of those wonderful situations where really placing the patient’s needs at the centre of the trial not only helps the patient and their siblings. It also increases efficiency in clinical trials and accelerates the drug’s progress to market.